Human recombinant activated protein C for severe sepsis and septic shock in adult and paediatric patients

Sepsis and septic shock are major causes of death. Sepsis is a complex syndrome resulting from a presumed or known infection, and its pathogenesis involves interactions between inflammation and blood clotting pathways. This serious medical condition is characterized by an inflammatory response to an infection which can affect the whole body. Patients with sepsis may have developed the inflammatory response because of microbes in their blood, urine, lungs, skin, or other tissues. Severe sepsis can lead to multiple organ failure due to blood clotting in the finer blood vessels. This reduces the amount of blood reaching the organs and septic shock ensues. Protein C reduces the clotting process and a lack of protein C can lead to an exaggeration of blood clotting. Sepsis and septic shock decrease protein C levels in the body. It has been suggested that human recombinant activated protein C (APC) will increase the levels of protein C and ameliorate or prevent multiple organ failure. In this updated Cochrane review we searched the databases until June 2012. We included six randomized clinical trials which involved 6781 people (6307 adult and 474 paediatric participants) with either a high or low risk of death. All trials had high risk of bias and were sponsored by the pharmaceutical industry (Eli Lilly). We found no evidence suggesting that APC reduced the risk of death in adults or children with severe sepsis or septic shock. On the contrary, APC increased the risk of serious bleeding.

On 25th October 2011, the European Medicines Agency issued a press release on the worldwide withdrawal of Xigris® (human recombinant activated protein C) from the market by Eli Lilly due to lack of beneficial effect on 28-day mortality in the PROWESS-SHOCK trial. Furthermore, Eli Lily has announced the discontinuation of all ongoing clinical trials. APC should not be used for sepsis or septic shock outside randomized clinical trials.

Current evidence does not support the use of human recombinant activated protein C in adults or children with severe sepsis or septic shock; moreover, there is an increased risk of bleeding associated with its use.

Authors' conclusions: 

This updated review found no evidence suggesting that APC should be used for treating patients with severe sepsis or septic shock. APC seems to be associated with a higher risk of bleeding. The drug company behind APC, Eli Lilly, has announced the discontinuation of all ongoing clinical trials using this drug for treating patients with severe sepsis or septic shock. APC should not be used for sepsis or septic shock outside randomized clinical trials.

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Background: 

Sepsis is a common and frequently fatal condition. Human recombinant activated protein C (APC) has been introduced to reduce the high risk of death associated with severe sepsis or septic shock. This systematic review is an update of a Cochrane review originally published in 2007.

Objectives: 

We assessed the benefits and harms of APC for patients with severe sepsis or septic shock.

Search strategy: 

We searched CENTRAL (The Cochrane Library 2013, Issue 5); MEDLINE (June 2012 to May 2013); EMBASE (June 2012 to May 2013); BIOSIS (June 2012 to May 2013); CINAHL (June 2012 to May 2013) and LILACS (June 2012 to May 2013). There was no language restriction.

Selection criteria: 

We included randomized clinical trials assessing the effects of APC for severe sepsis or septic shock in adults and children. We excluded studies on neonates. We considered all-cause mortality at day 28 and at the end of study follow up, and hospital mortality as the primary outcomes.

Data collection and analysis: 

We independently performed trial selection, risk of bias assessment, and data extraction in duplicate. We estimated relative risks (RR) for dichotomous outcomes. We measured statistical heterogeneity using the I2 statistic. We used a random-effects model.

Main results: 

We identified one new randomized clinical trial in this update which includes six randomized clinical trials involving 6781 participants in total, five randomized clinical trials in adult (N = 6307) and one randomized clinical trial in paediatric (N = 474) participants. All trials had high risk of bias and were sponsored by the pharmaceutical industry. APC compared with placebo did not significantly affect all-cause mortality at day 28 compared with placebo (856/3643 (23.49%) versus 837/3549 (23.58%); RR 1.00, 95% confidence interval (CI) 0.88 to 1.14; I2 = 49%). APC did not significantly affect in-hospital mortality (393/1767 (22.2%) versus 379/1710 (22.1%); RR 1.01, 95% CI 0.87 to 1.16; I2 = 20%). APC was associated with an increased risk of serious bleeding (113/3424 (3.3%) versus 74/3343 (2.2%); RR 1.45, 95% CI 1.08 to 1.94; I2 = 0%). APC did not significantly affect serious adverse events (463/3334 (13.9%) versus 439/3302 (13.2%); RR 1.04, 95% CI 0.92 to 1.18; I2 = 0%). Trial sequential analyses showed that more trials do not seem to be needed for reliable conclusions regarding these outcomes.